Andembry approved in Japan as preventive treatment for HAE
Approval based on VANGUARD study, which saw 85% attack rate reduction
Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved Andembry (garadacimab) as a treatment to reduce the risk of swelling attacks in people with hereditary angioedema (HAE).
The approval makes Andembry the first long-term preventive treatment in Japan available in a prefilled pen and given once a month by injection under-the-skin, according to its developer CSL Behring.
“Andembry represents a major advancement in the management of hereditary angioedema, offering people living with this life-threatening condition long-term disease control through a patient-centric and convenient administration method,” Bill Mezzanotte, MD, executive vice president and head of research and development at CSL, said in a company press release.
The approval in Japan comes less than a month after Andembry was approved for HAE in the U.K., in Australia, and the European Union. Its approval is being considered in the U.S.
“This milestone is the result of decades of dedication, and we extend our gratitude to the colleagues, physicians, and patients who made this possible for HAE patients and CSL,” Mezzanotte said.
‘Breakthrough therapy’ for HAE
HAE is a genetic disease where swelling attacks are driven by the overproduction of bradykinin, a signaling molecule. An antibody-based therapy, Andembry inhibits factor XII (FXII), a protein that’s part of the chain of events that leads to bradykinin’s production. By blocking FXII, Andembry shooulld bring bradykinin levels down, reducing the risk of swelling attacks.
“Andembry is a breakthrough therapy as the first and only treatment targeting activated Factor XII, the key initiator of HAE attacks,” said Rose Fida, PhD, executive director and regional lead of CSL research and development in Japan and China. “With its novel mechanism, once-monthly subcutaneous dosing and easy-to-use prefilled pen, Andembry is set to transform the way HAE is managed in Japan.”
CSL’s applications for Andembry’s approval were based mainly on data from VANGUARD (NCT04656418), a Phase 3 clinical trial that enrolled 64 people with HAE, ages 12 and older who were randomly assigned to monthly injections of Andembry or a placebo for about six months.
The rate of swelling attacks was reduced by more than 85% in patients given Andembry over those given a placebo, results showed. About two-thirds of those given Andembry were completely free from swelling attacks throughout the trial.
Most patients who completed VANGUARD enrolled in an open-label extension study (NCT04739059) wherein all the participants are being treated with Andembry and monitored for long-term safety and efficacy outcomes. The extension study is still ongoing, but available data have consistently indicated the treatment can help prevent swelling attacks.
A separate Phase 3 trial (NCT05819775) is testing Andembry in children with HAE ages 2-11. That study is expected to conclude in late 2026.
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